A Randomized Trial of Thymoglobulin to Prevent Chronic Graft Versus Host Disease in Patients Undergoing Hematopoietic Progenitor Cell Transplantation (HPCT) From Unrelated Donors
This study is a non-blinded, randomized, multicentre trial testing the effect of
Thymoglobulin® vs. placebo on the primary outcome of cGVHD. Subjects will be children and
adults having unrelated donor transplants.
Intervention: Infusion of Thymoglobulin® on three days prior to the transplant.
Hypothesis: The hypothesis is that the use of Thymoglobulin® in the experimental group will
result in an absolute 20% increase in the number of patients free of cGVHD at 12 months, the
time of peak incidence, from 20% in the control group to 40% in the experimental group.
Outcome Measures: The Primary Outcome Measure is freedom from cGVHD at 12 months from
transplantation, defined as withdrawal of all systemic immunosuppressive agents and without
resumption up to 12 months (a binary end-point, yes/no). Secondary outcome measures:
Quality of Life, overall incidence of cGVHD (including untreated cases and resolved cases),
the incidence of "extensive" cGVHD, time to non-relapse mortality, time to all-cause
mortality, time to relapse of leukemia, graft rejection or failure (Yes vs. No), serious
infection (Yes vs. No), CMV activation (Yes vs. No), organ-specific grading of chronic graft
versus host disease, resumption of immunosuppressive agents after 12 months (Yes vs. No),
doses of immunosuppressive drugs still required at 12 months, and incidence of acute graft
versus host disease.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Prevention
Freedom from Chronic GVHD
"Freedom from Chronic GVHD" is defined as withdrawal of all systemic immunosuppressive agents and without resumption up to 12 months (a binary endpoint, yes/no)
12 months post transplant
Irwin Walker, MD
McMaster University, Faculty of Health Sciences
Canada: Health Canada